Advocacy
Commitment to Fight for MSD Families
In the United States, a disease is defined as rare when it affects fewer than 200,000 people. More than 30 million Americans or 1 in 10 live with one or more rare diseases. That makes rare diseases more common than we may think.
Unfortunately, we also know that rare disease patients wait an average of 6.3 years after symptoms present before receiving a confirmed diagnosis, having received several incorrect diagnoses during that time. Even after a diagnosis, it doesn’t mean you have a treatment waiting for you. Currently, 93% – 95% of the more than 10,000 known rare diseases have no U.S. Food and Drug Administration-approved therapies.
That is why it’s important for the United MSD Foundation to be active in advocacy.
We are committed to ensuring patients have a place at the table in policy and access discussions, research funding is increased for rare diseases, the development approval of drugs and treatments is accelerated, and ethical implications are considered during decision-making by our policymakers and industry partners.
The United MSD Foundation is concentrated on advancing newborn screening for lysosomal storage disorders, including MSD, as early diagnosis is critical for early intervention, potential involvement in clinical trials, and ongoing support of families until therapies are approved.
If you would like to be involved in our advocacy efforts, please contact our Executive Director.
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